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GUIDANCE DOCUMENT

Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations Draft Guidance for Industry December 2021

Download the Draft Guidance Document Read the Federal Register Notice
Draft Level 1 Guidance

Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

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Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the FDA considers your comment on a draft guidance before it begins work on the final version of the guidance, submit either online or written comments on the draft guidance before the close date.

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2021-D-1140

Docket Number:
FDA-2021-D-1140
Issued by:
Guidance Issuing Office
Center for Drug Evaluation and Research

This guidance is intended for sponsor-investigators (hereafter referred to as sponsors) developing individualized investigational antisense oligonucleotide (ASO) drug products for a severely debilitating or life-threatening (SDLT) genetic disease. Most often, individuals with such diseases will have no alternative treatment options, and their diseases will be rapidly progressing, resulting in early death and/or devastating or irreversible morbidity within a short time frame without treatment. In these situations, drug development targeted to a larger number of patients with the same disease is not anticipated because of the specificity of the mechanism of action of the ASO combined with the rarity of the treatment-amenable patient population. The gene variant or variants that are targeted by the ASO drug product should be unique to the trial participant(s) and generally only reported in a small number of patients (typically 1 to 2) in the disease population. If more than a few patients may be candidates for targeted treatment with the ASO drug product, then the ASO is no longer considered individualized, and the sponsor should discuss a drug development plan of the investigational ASO drug product for a larger patient population with the relevant review division.

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